1. Katalin,Karikó 匈牙利 Szeged University (Hungary) 2022美国医学院院士 ;2021拉斯克奖;2023诺贝尔生理学或医学奖;2025美国工程院院士;2025美国科学院院士 ASL mRNA-LNP Therapeutic for the Treatment of Argininosuccinic Aciduria Enables Survival Benefit in ..
2. Jennifer A,Doudna 美国 University of California-Berkeley 2010美国医学院院士;2002美国科学院院士;2020沃尔夫医学奖;2020诺贝尔化学奖 CRISPR-Cpf1 mediates efficient homology-directed repair and temperature-controlled genome editing
3. Etienne-Emile,Baulieu 法国 UNIVERSITE PARIS SACLAY 1990美国科学院院士;1989拉斯克奖 A decrease in Fkbp52 alters autophagosome maturation and A152T-tau clearance in vivo
4. Trudy F. C,Mackay 美国 Clemson University 2010美国科学院院士;2016沃尔夫农业奖 Ex vivo gene editing and cell therapy for hereditary tyrosinemia type 1
5. Nahum Sonenberg 加拿大 McGill University, Canada 2015美国医学院院士;2015美国科学院院士;2014沃尔夫医学奖 Clinical, neuroradiological, and molecular characterization of mitochondrial threonyl-tRNA- syntheta..
6. Kazutoshi,Mori 日本 Kyoto University 2014拉斯克奖 A Collection of Transgenic Medaka Strains for Efficient Site-Directed Transgenesis Mediated by phiC3..
7. Napoleone,Ferrara 美国 University of California San Diego 2006美国医学院院士；2006美国科学院院士;2010拉斯克奖 Consensus guidelines for the use and interpretation of angiogenesis assays
8. Ralf,Bartenschlager 德国 University of Heidelberg 2016拉斯克奖 TALEN/CRISPR-mediated engineering of a promoterless anti-viral RNAi hairpin into an endogenous miRNA..
9. Gahl, William A. 美国 National Human Genome Research Institute 2018美国医学院院士 Insights into the renal pathophysiology in Hermansky-Pudlak syndrome-1 from urinary extracellular ve..
10. Marianne E,Bronner 美国 California Institute of Technology 2015美国科学院院士 Generation of a zebrafish neurofibromatosis model via inducible knockout of nf2a/b
11. Lupski, James R. 美国 Baylor College of Medicine 2002美国医学院院士 Homozygous variants in WDR83OS lead to a neurodevelopmental disorder with hypercholanemia
12. Lupski, James R. 美国 Baylor College of Medicine 2002美国医学院院士 Loss of symmetric cell division of apical neural progenitors drives DENND5A-related developme..
13. Gleeson, Joseph G. 美国 University of California, San Diego 2013美国医学院院士 Loss of symmetric cell division of apical neural progenitors drives DENND5A-related developme..
14. Richard L. Huganir 美国 Johns Hopkins University 2011美国医学院院士;2004美国科学院院士 Context-dependent hyperactivity in syngap1a and syngap1b zebrafish models of SYNGAP1-r..
15. Cavazzana, Marina 法国 Paris University Medical School 2019美国医学院院士 Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cell..
16. Scott W. Lowe 美国 Memorial Sloan Kettering Cancer Center 2017美国科学院院士;2019美国医学院院士 Engineering megabase-sized genomic deletions with MACHETE[Molecular Alteration of Chromosomes with E..
17. Katherine A. High 美国 AskBio 2007美国医学院院士;2021美国科学院院士 Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec
18. Lupski, James R. 美国 Baylor College of Medicine 2002美国医学院院士 Bi-allelic ACBD6 variants lead to a neurodevelopmental syndrome with progressive and complex movemen..
19. Gleeson, Joseph G. 美国 University of California, San Diego 2013美国医学院院士 Bi-allelic ACBD6 variants lead to a neurodevelopmental syndrome with progressive and complex movemen..
20. Kinoshita, Taroh 日本 Osaka University 2025美国科学院院士 AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse models